Rescue of common and rare exon 2 skipping variants of the GAA gene using modified U1 snRNA
利用修饰的U1 snRNA拯救GAA基因常见和罕见的第2外显子跳跃变异体
期刊:Molecular Medicine
影响因子:6.4
doi:10.1186/s10020-025-01090-z
Peruzzo, Paolo; Bergamin, Natascha; Bon, Martina; Cappelli, Sara; Longo, Alessandra; Goina, Elisa; Stuani, Cristiana; Buratti, Emanuele; Dardis, Andrea